Nonviral Vectors for Gene Therapy


Book Description

Gene transfer within humans has been an obstacle until about 10 years ago. At that time, it was found that viral vectors were effective carriers of "healthy genes" into patients' cells. The problem, however, was that viral vectors proved unnecessarily harmful to humans: subjects experienced inflamatory activity and negative immunological responses to the genes. Viral vectors were also unable to meet the needs of the pharmaceutical community: they were not reproducible in large-scale proportions in cost-effective ways.Thus, research was undertaken to find a safer way to transfer genes to patients without jeopardizing the safety of the patient. And so non-viral vectors were discovered. This volume presents the various non-viral vectors currently under development. Although not methodologically oriented, it will provide the necessary details behind the development of the vectors. This information will prove useful to both researchers and clinicians.Key Features* Presents state-of-the art developments of nonviral vectors as tools for modern molecular medicine* Covers all types of nonviral vectors, from molecular structure to therapeutic applicationProvides a comprehensive review of synthetic vectors* Includes contributions from major investigators and leading experts in the field




Polymers and Nanomaterials for Gene Therapy


Book Description

Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy. The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy. This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy. - Explores current challenges in the research of genetic diseases - Discusses polymers for gene therapy and their function in designing advanced materials - Provides examples of organic and inorganic nanomaterials for gene therapy - Includes labeling, targeting, and assays - Looks at characterization, physico-(bio)chemical properties, and applications




Polymeric Gene Delivery Systems


Book Description

​The series Topics in Current Chemistry Collections presents critical reviews from the journal Topics in Current Chemistry organized in topical volumes. The scope of coverage is all areas of chemical science including the interfaces with related disciplines such as biology, medicine and materials science. The goal of each thematic volume is to give the non-specialist reader, whether in academia or industry, a comprehensive insight into an area where new research is emerging which is of interest to a larger scientific audience. Each review within the volume critically surveys one aspect of that topic and places it within the context of the volume as a whole. The most significant developments of the last 5 to 10 years are presented using selected examples to illustrate the principles discussed. The coverage is not intended to be an exhaustive summary of the field or include large quantities of data, but should rather be conceptual, concentrating on the methodological thinking that will allow the non-specialist reader to understand the information presented. Contributions also offer an outlook on potential future developments in the field. The chapter "Polymeric Nanoparticle-Mediated Gene Delivery for Lung Cancer Treatment" is available open access under a Creative Commons Attribution 4.0 International License via link.springer.com.




Gene and Cell Therapy: Biology and Applications


Book Description

Recent advances in stem cell biology, nanotechnology and gene therapy have opened new avenues for therapeutics. The availability of molecular therapeutics that rely on the delivery of DNA, RNA or proteins, harnessing enhanced delivery with nanoparticles, and the regenerative potential of stem cells (adult, embryonic or induced pluripotent stem cells) has had a tremendous impact on translational medicine. The chapters in this book cover a range of strategies for molecular and cellular therapies for human disease, their advantages, and central challenges to their widespread application. Potential solutions to these issues are also discussed in detail. Further, the book addresses numerous advances in the field of molecular therapeutics that will be of interest to the general scientific community. Lastly, the book provides specific examples of disease conditions for which these strategies have been transferred to the clinic. As such, it will be extremely useful for all students, researchers and clinicians working in the field of translational medicine and molecular therapeutics.




Immunopharmacology


Book Description

During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.




Synthetic DNA Delivery Systems


Book Description

DNA delivery into cells is a rapidly developing area in gene therapy and biotechnology. Moreover, it is a powerful research tool to determine gene structure, regulation, and function. Viral methods of DNA delivery are well-characterized and efficient, but little is known about the toxicity and immunogenecity of viral vectors. As a result, non-viral, transfection methods of DNA delivery are of increasing interest. Synthetic DNA Delivery Systems is a comprehensive and current resource on DNA transfection. The use of histidine-rich peptides and polypeptides as DNA delivery systems and self-assembled delivery systems based on cationic lipids and polymers are discussed. Targeted delivery to organelles, tumor cells and dendritic cells comprise an important topic.




Non-viral Gene Therapy


Book Description

Presents information on non-viral gene-delivery techniques, covering a spectrum of disciplines that include chemistry, molecular biology, cell biology, and pharmacokinetics. This work is useful to researchers and engineers in genetic engineering, molecular medicine, biochemical engineering, and biotechnology.




Metallosurfactants


Book Description

Metallosurfactants Provides up-to-date coverage of the synthesis, properties, and applications of metallosurfactants Metallosurfactants: From Fundamentals to Catalytic and Biomedical Applications is a thorough introduction to amphiphilic compounds that allow to incorporate metal ions in the surfactant system. This comprehensive reference and guide describes the fundamentals of metal surfactant complexes, highlights recent advances in the field, and explores current and future applications and research areas. Gradually progressing from basic to advanced topics, the authors first explain the classification and characterization of metallosurfactants before delving into more complex concepts and various catalytic, sensing, and biomedical applications. The book begins with coverage of the synthesis of metallosurfactants and their surface, interfacial, and aggregation behavior. Subsequent chapters discuss applications of metallosurfactants in areas such as drug delivery, molecular machines, transfection, nanoparticle synthesis, and carbon monoxide-releasing molecules (CORMs). Other topics include the use of metallosurfactants as catalysts in organic reactions, and as anticancer and antimicrobial agents in drug delivery and formulation. This unique reference Provides an overview of the structure-function relationship, synthesis methods, and characterization of metallosurfactants Reviews current trends in metallosurfactant development and research Examines the use of metallosurfactants in a wide range of reactions, including esterolytic reactions and hydrogen generation Discusses advanced applications of metallosurfactants, e.g. as nanoreactors for nanoparticle synthesis, non-viral transfection vectors, and sensors Metallosurfactants: From Fundamentals to Catalytic and Biomedical Applications is an excellent introduction to the growing field of metallosurfactant chemistry as well as a concise, highly useful reference for researchers and scientists in both academia and industry.




Muscle Gene Therapy


Book Description

Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.




Novel Gene Therapy Approaches


Book Description

Gene therapy has the potential to be a tailor-made therapeutic with increased specificity and decreased side effects that can offer a "cure" for many disorders. The aim of this book is to provide up-to-date reviews of the rapidly growing field of gene therapy. Chapters cover a large range of topics including methods of gene delivery, and identification of targets with several papers on cancer gene therapy. If more people become aware of the true nature and potential of gene therapy, perhaps we can achieve the full benefit of such an innovative approach for the treatment of a range of diseases, including cancer.